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University of Rochester

601 Elmwood Avenue
Rochester, NY 14642
United States

A third of babies with Down Syndrome are born with an abnormal blood production called TAM (transient abnormal myelopoiesis), which is an expansion of immature, leukemia-like, blood cells that carry a mutation called Gata1s. TAM is associated with a significant increase in death shortly after birth, and roughly 1 in 4 babies that survive the neonatal period develops leukemia before the age of 4. It is not well understood how or when TAM arises during fetal life.

B-lymphoblastic leukemia (B-ALL) is the most common childhood cancer and has an excellent overall chance of cure. However, infants younger than 12 months old at diagnosis are the exception to this success. Regardless of their more intense treatment, infants with B-ALL are far more likely than older children to experience leukemia relapse. We must improve our ability to predict which infants are destined to relapse so that we can introduce new treatment approaches for those most in need.

Background

Background

Acute lymphoblastic leukemia (ALL) is the most common form of childhood cancer and relapsed ALL is the most common cause of death from childhood cancer. Exciting new research has shown that leukemia cells depend their survival on nonmalignant, noncancerous cells in the body.

Background

Leukemia is the most common form of cancer among children and adolescents, and approximately 2,000 infants within their first year of life developed life threatening acute leukemia in the United States. The presence of the MLL (Mixed Lineage Leukemia) gene translocation is the most significant independent factor associated with poor outcome and high risk of relapse in infant leukemia.

With chemotherapy treatment, 80-90% of children with acute myeloid leukemia (AML) achieve remission. However, 30-40% of these patients subsequently suffer recurrence, and the long-term survival rate is only 50%. Novel therapies are urgently needed to prevent or treat recurring AML. Parthenolide, a naturally-occurring compound, induces robust AML cell killing and has the potential to reduce recurrence of AML by killing cells resistant to traditional chemotherapeutics and responsible for relapse.

The most common childhood cancer is acute lymphoblastic leukemia. While three-quarters of these children are cured, about one-quarter relapse. The prognosis for these children is poor without very aggressive treatment that includes bone marrow transplantation. Even with transplant the risk of relapse is still high. Doctors and scientists do not fully understand the reasons the leukemia comes back after transplant.